Sarepta Therapeutics said on Wednesday it would lay off 36% of its workforce, or about 500 employees, after the recent deaths of two patients who had received its gene therapy Elevidys. Its shares ...

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.

Sarepta Therapeutics faces a challenging period as it navigates recent setbacks with its groundbreaking gene therapy, leading ...

CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

The Centers for Medicare and Medicaid Services has named 33 states, as well as Washington, D.C., and Puerto Rico, as participants in its Cell and Gene Therapy Access Model, which is designed to treat ...

The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...

Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy (GA), a complication of age-related macular degeneration (AMD), as it prepares to test it in a clinical ...

Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...

Sarepta Therapeutics said on Wednesday it would lay off 36% of its workforce, or about 500 employees, after two recent deaths of patients who had received its gene therapy, Elevidys. Shares of the ...